Vosoritide

Also known as: BMN 111 · Voxzogo

FDA-approved

Last verified: October 30, 2026

At a glance

FDA approval FDA-approved 1 application
Randomized controlled trials 7 of 133 publications on PubMed
Registered clinical trials 18 12 active
Adverse event reports 1,331 369 classified as serious
  • Vosoritide is an FDA-approved drug, marketed as Voxzogo (first approval: November 19, 2021).
  • PubMed lists 133 publications mentioning it, including 7 randomized controlled trials and 5 animal-only studies.
  • ClinicalTrials.gov lists 18 registered studies testing it (12 active).
  • FDA's adverse event system (FAERS) holds 1,331 reports mentioning it; 369 classified as serious. Reports do not prove causation.

Research topics

  • Achondroplasia 65
  • Natriuretic Peptide, C-Type 48
  • Receptor, Fibroblast Growth Factor, Type 3 19
  • Body Height 16
  • Bone and Bones 9
  • Quality of Life 9
  • Mutation 8
  • Growth Disorders 7
  • Human Growth Hormone 7
  • Dwarfism 6

Most frequent MeSH subject headings in the latest PubMed records (English, as indexed).

Regulatory status

Drugs@FDA applications

Application Product Status First approval Sponsor
NDA214938 VOXZOGO Approved, marketed November 19, 2021 BIOMARIN PHARM

Source: Drugs@FDA (openFDA) · verified October 30, 2026 · API query

FDA compounding safety list

Not on FDA's list of bulk drug substances that may present significant safety risks in compounding.

Source: FDA.gov — Category 2 bulk drug substances · verified October 30, 2026

Removal of a substance from a regulatory list does not mean it has been approved. A "research use only" label does not make a product safe or legal for human use.

Scientific publications

  • All publications 133
  • Human (MeSH) 79
  • Animal only 5
  • Clinical trials 11
  • Randomized controlled trials 7
  • Systematic reviews & meta-analyses 4
  • Case reports 9
  • Retracted 0

Counts are PubMed records that mention the peptide in the title or abstract. Categories overlap, and a mention is not the same as testing the peptide as a treatment. Each number opens the exact PubMed search. Effects seen in laboratory or animal studies may not occur in humans. A registered clinical trial does not show that a substance is effective or safe.

Latest publications

Source: PubMed (NCBI) · verified October 30, 2026

Clinical trials

Registered
18
Active
12
With posted results
3

Conditions studied

  • Achondroplasia 10
  • Hypochondroplasia 3
  • Short Stature 2
  • Noonan Syndrome 1
  • Idiopathic Short Stature 1
  • MPS IVA 1
  • MPS VI 1
  • Turner Syndrome 1
Study Status Phase Start
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT07441876 BioMarin Pharmaceutical
Recruiting Phase 2, Phase 3 April 20, 2026
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
NCT07126262 BioMarin Pharmaceutical
Recruiting Phase 2 July 30, 2025
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT07073014 BioMarin Pharmaceutical
Enrolling by invitation Phase 3 June 20, 2025
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
NCT06668805 BioMarin Pharmaceutical
Active, not recruiting Phase 2 November 22, 2024
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
NCT06382155 BioMarin Pharmaceutical
Recruiting Phase 2 October 21, 2024
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT06455059 BioMarin Pharmaceutical
Active, not recruiting Phase 3 June 17, 2024
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
NCT05845749 University of California, San Francisco
Active, not recruiting Phase 1, Phase 2 September 25, 2023
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT04554940 BioMarin Pharmaceutical
Active, not recruiting Phase 2 October 10, 2020
Vosoritide for Selected Genetic Causes of Short Stature
NCT04219007 Andrew Dauber
Active, not recruiting Phase 2 August 4, 2020
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT03989947 BioMarin Pharmaceutical
Active, not recruiting Phase 2 June 12, 2019
Show 8 more studies
Study Status Phase Start
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03424018 BioMarin Pharmaceutical
Active, not recruiting Phase 3 December 12, 2017
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT02724228 BioMarin Pharmaceutical
Active, not recruiting Phase 2 January 26, 2016
Vosoritide for Short Stature in Turner Syndrome
NCT05849389 Roopa Kanakatti Shankar, MBBS, MS
Unknown status Phase 2 April 12, 2024
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT05813314 BioMarin Pharmaceutical
Terminated Phase 1 March 15, 2023
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT03583697 BioMarin Pharmaceutical Results posted
Completed Phase 2 June 13, 2018
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03197766 BioMarin Pharmaceutical Results posted
Completed Phase 3 December 12, 2016
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT02055157 BioMarin Pharmaceutical Results posted
Completed Phase 2 January 13, 2014
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT01590446 BioMarin Pharmaceutical
Completed Phase 1 Feb 2012

All 18 studies on ClinicalTrials.gov ↗

Source: ClinicalTrials.gov · verified October 30, 2026

Adverse event reports

Reports
1,331
Serious
369
With death outcome
8

Most reported reactions

  • Injection site pain 179
  • Injection site erythema 103
  • Pyrexia 78
  • Injection site reaction 72
  • Vomiting 65
  • Drug ineffective 62
  • Injection site swelling 55
  • Foramen magnum stenosis 40
  • Ear infection 38
  • Injection site bruising 33
  • Off label use 33
  • Headache 29

Adverse event reports do not prove that the substance caused these effects. Side effects not listed here may still occur. Reaction terms are MedDRA terms as reported to FAERS.

Source: FDA FAERS (FAERS data as of July 30, 2026) · verified October 30, 2026 · API query

FDA prescribing information

Official US prescribing information. Dosing sections are intentionally not shown.

Source: DailyMed / openFDA

Change history

No changes since monitoring began on October 30, 2026.